Adelphi Values, Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany.
J Patient Rep Outcomes. 2024 Aug 15;8(1):97. doi: 10.1186/s41687-024-00776-y.
Breast cancer is one of the most common cancers in women. Patient-reported outcome measures are used to evaluate patients' health-related quality of life in clinical breast cancer studies. This study evaluated the structure, validity, reliability, and responsiveness of the National Comprehensive Cancer Network-Functional Assessment of Cancer Therapy-Breast Cancer Symptom Index (NFBSI-16) subscales in a clinical trial featuring patients with advanced/metastatic breast cancer (aBC), and estimated NFBSI-16 meaningful change thresholds.
Data from 101 patients with aBC enrolled in a phase II trial (Xenera-1) were included for psychometric evaluation of the NFBSI-16. Subscale structure was evaluated by assessing inter-item correlations, item-total correlations, and internal consistency (cycles 2 and 5). Validity was assessed using scale-level convergent validity (cycles 2 and 5) and known-groups (Baseline). Reliability was analysed via test-retest at cycles 3-4, and responsiveness to improvement and worsening was evaluated at cycles 5, 7, and 9. Meaningful change thresholds were estimated using anchor-based methods (supported by distribution-based methods) at cycles 5, 7, and 9.
NFBSI-16 internal consistency was acceptable, but item-total correlations suggested that its subscales and the GP5 item (side-effect of treatment) scores may be preferred over a total score. Convergent and known-groups evidence supported NFBSI-16 validity. Test-retest reliability was good to excellent for Total and DRS-P (disease-related symptoms: physical) scales, and moderate for the GP5 item. Responsiveness to worsening was generally demonstrated, but responsiveness to improvement could not be demonstrated due to limited observed improvement. Anchor-based meaningful change thresholds were estimated for DRS-P and Total scores.
This study provides evidence that the NFBSI-16 has desirable psychometric properties for use in clinical studies in aBC. It also provides estimates of group- and individual-level meaningful change thresholds to facilitate score interpretation in future aBC research.
乳腺癌是女性最常见的癌症之一。患者报告的结局测量被用于评估临床乳腺癌研究中患者的健康相关生活质量。本研究评估了国家综合癌症网络-癌症治疗功能评估-乳腺癌症状指数（NFBSI-16）在晚期/转移性乳腺癌（aBC）临床试验中的结构、有效性、可靠性和反应性，并估计了 NFBSI-16 的有意义变化阈值。
纳入了 101 例 aBC 患者的 Xenera-1 期临床试验数据，用于 NFBSI-16 的心理测量评估。通过评估项目间相关性、项目总分相关性和内部一致性（第 2 周期和第 5 周期）来评估子量表的结构。通过第 2 周期和第 5 周期的量表水平收敛效度和已知组（基线）评估有效性。通过第 3-4 周期的测试-重测分析评估可靠性，并在第 5、7 和 9 周期评估对改善和恶化的反应性。通过第 5、7 和 9 周期的基于锚的方法（辅以基于分布的方法）估计有意义的变化阈值。
NFBSI-16 的内部一致性是可以接受的，但项目总分相关性表明，其子量表和 GP5 项目（治疗副作用）的得分可能优于总分。NFBSI-16 的有效性得到了收敛性和已知组的证据支持。总评分和 DRS-P（与疾病相关的症状：身体）量表的测试-重测可靠性良好至优秀，GP5 项目的可靠性为中等。通常显示出对恶化的反应性，但由于观察到的改善有限，无法证明对改善的反应性。为 DRS-P 和总评分估计了基于锚的有意义的变化阈值。
本研究为 NFBSI-16 在 aBC 的临床研究中具有理想的心理测量特性提供了证据。它还提供了组和个体水平有意义的变化阈值的估计值，以促进未来 aBC 研究中对分数的解释。
